Aptorum Group Announces Significant Progress of Repurposed Drug Candidate, SACT-1 for Neuroblastoma Targeting IND Submission in H2 2020
Aptorum Group Limited (Nasdaq: APM) (“Aptorum Group”), a biopharmaceutical company focused on the development of novel therapeutics to address global unmet medical needs, announces positive data and development in relation to its repurposed drug candidate, SACT-1, for the treatment of neuroblastoma, a rare type of childhood cancer that develops in infants and young children. Subject to completion of current validation studies, Aptorum Group plans to leverage the 505(b)(2) pathway and submit an IND submission with the FDA for SACT-1 in H2 2020.1
SACT-1 is the first repurposed drug candidate to be developed under the Smart-ACTTM drug discovery platform, which employs a systematic approach to identify, repurpose and develop existing approved drugs against a currently identified universe of 7000+ (and increasing) orphan diseases.2
Through this platform, Aptorum Group intends to accelerate and fast track repurposed drug candidates, which usually have well established human safety and toxicity profiles and data, through the development and clinical phases in order to address the rapidly growing market of orphan diseases. Aptorum Group aims to screen a number of orphan disease areas including, but not limited to, oncology, autoimmune, metabolic and genetic diseases.
Through the Smart-ACTTM platform, Aptorum has successfully identified potential efficacy for and develops SACT-1 for the treatment of neuroblastoma, being an entirely new therapeutic area from its approved indication. In our recent studies, SACT-1 has been shown to be effective against numerous neuroblastoma cell lines, of which 2 are MYCN-amplified cells, which represent the high-risk neuroblastoma patient group. In addition, by using a combination index as a quantitative measure of the extent of drug interaction, Aptorum Group has seen a high and robust synergism between SACT-1 and traditional chemotherapy in vitro, indicating a potential efficacy enhancement/dose reduction of the chemotherapy. In addition, in our recent study, the maximum tolerable dose of SACT-1 in a rodent model was determined to be higher than 400mg/kg. Compared with the MTD of standard chemotherapy such as paclitaxel (20-30mg/kg)3 and cisplatin (6mg/kg) 4, the safety profile of SACT-1 appears to be very impressive.
The reformulation of SACT-1 is a pediatric formulation to better address the needs of neuroblastoma patients who are exclusively children younger than 5. Based on our internal observations of pre-existing information from approved products,5 SACT-1 also exhibits a well-established safety profile: at 150mg/day, the death rate was 0% in prior clinical studies) with no dosage related adverse events.
Neuroblastoma is a rare form of cancer, and classified as an orphan disease, that forms in certain types of nerve tissue and most frequently in the adrenal glands as well as spine, chest, abdomen or neck, predominantly in children, especially for those aged 5 years and below. For the high-risk group, which is close to 20%6 of total new patient population per year, the 5-year survival rate of this condition is around 40-50% as observed by the American Cancer Society7. The current high drug treatment cost for high risk patients can average USD200,000 per regimen (all 6 cycles)8. In addition, most pediatric patients often do not tolerate or survive the relevant chemotherapy stage which, subject to further clinical studies, may be positively addressed by the SACT-1 candidate due to the potential synergistic effects when applied with standard chemotherapy as described above.
For further general presentation, please visit: http://ir.aptorumgroup.com/static-files/bcf77574-7bd6-4b9d-8110-d53837238f16
For further technical presentation, please visit: http://ir.aptorumgroup.com/static-files/66346f79-7a03-474a-89be-0eaafaa00d9d
About Aptorum Group Limited
Aptorum Group Limited (Nasdaq: APM) is a pharmaceutical company dedicated to developing and commercializing novel therapeutics to tackle unmet medical needs. Aptorum Group is pursuing therapeutic projects in orphan diseases, infectious diseases, metabolic diseases and other disease areas.
For more information about Aptorum Group, please visit www.aptorumgroup.com.
About Smart Pharma Group
Smart Pharma Group is wholly owned by Aptorum Group Limited. Smart Pharma Group focuses on systematically identifying and repurposing existing approved drugs for the treatment of a large array of orphan diseases. Smart Pharma Group conducts both computational based screening and clinical validations in advancing the development of its repurposed candidates.
Disclaimer and Forward-Looking Statements
This press release includes statements concerning Aptorum Group Limited and its future expectations, plans and prospects that constitute “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. For this purpose, any statements contained herein that are not statements of historical fact may be deemed to be forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “should,” “expects,” “plans,” “anticipates,” “could,” “intends,” “target,” “projects,” “contemplates,” “believes,” “estimates,” “predicts,” “potential,” or “continue,” or the negative of these terms or other similar expressions. Aptorum Group has based these forward-looking statements, which include statements regarding projected timelines for application submissions and trials, largely on its current expectations and projections about future events and trends that it believes may affect its business, financial condition and results of operations. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions including, without limitation, risks related to its announced management and organizational changes, the continued service and availability of key personnel, its ability to expand its product assortments by offering additional products for additional consumer segments, development results, the company’s anticipated growth strategies, anticipated trends and challenges in its business, and its expectations regarding, and the stability of, its supply chain, and the risks more fully described in Aptorum Group’s Form 20-F and other filings that Aptorum Group may make with the SEC in the future. As a result, the projections included in such forward-looking statements are subject to change. Aptorum Group assumes no obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.
1 If the FDA deems the 505(b)(2) pathway as an acceptable route for approval of SACT-1, the Company will be able to leverage existing clinical and nonclinical data in conjunction with sponsor-initiated studies to accelerate development and approval of SACT-1.
2 See https://rarediseases.info.nih.gov/diseases/pages/31/faqs-about-rare-diseases
3 Clin Cancer Res. 5(11):3632-8.
4 BMC Cancer 17: 684 (2017).
5 Subject to FDA's approval and on a case-by-case basis, a 505(b)(2) application can rely in part on existing information from approved products (such as the FDA's previous findings on safety and efficacy) or products in literature (such as data available). However, typically speaking, the applicant is nonetheless required to carry out a Phase 1 bridging study to compare the Reference Listed Drug and reference the established safety and efficacy information.
6 Annu Rev Med. 2015; 66: 49–63.
About Business Wire
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
Nike Extends its Athletic Leadership5.2.2020 19:00:00 CET | Press release
Today, NIKE, Inc. (NYSE: NKE), unveiled breakthrough innovation for athletes competing in the Tokyo 2020 Games, including footwear designed to provide runners with a measurable performance benefit for a new era of competition. The NEXT% platform, introduced by barrier-breaking marathoner Eliud Kipchoge, will now expand into new disciplines following its unparalleled success in distance running. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200205005708/en/ An international group of athletes joined the Nike 2020 Forum in New York to unveil the brand's latest performance and sustainable innovations. From left to right: Ibtihaj Muhammad, English Gardner, Leticia Bufoni, Blake Leeper, Timothy Cheruiyot, Dina Asher-Smith, Tomoya Ochiai, Sky Brown, Chris Mosier, Nyjah Huston, DeAnna Price, Bebe Vio, Kevin Mayer, Megan Blunk, Brandi Chastain, Aaron Brown, Sophie Hahn, Aori Nishimura, Diana Taurasi, Miles Chamley-Watson, Leon Schae
Cepton Expands With $50 Million Investment From Koito5.2.2020 18:00:00 CET | Press release
Cepton Technologies, Inc., a leading provider of state-of-the-art, intelligent, lidar-based solutions, announced today that it has raised over $50 million in Series C funding. This brings Cepton’s total funding to nearly $100 million, providing a strong foundation to substantially ramp up its R&D efforts, expand its footprint in the automotive market and support major customers worldwide. The latest funding round was led by Koito Manufacturing Co., Ltd. (TYO:7276), the automotive Tier 1 and world-leading provider of automotive lighting systems, with an investment of $50 million. Existing investors in Cepton also participated in this round. As part of the transaction, Koito will obtain non-exclusive rights to manufacture and sell Cepton’s lidar sensor design for an automotive application, using key components supplied by Cepton. Cepton will use the proceeds to accelerate development and deployment of its advanced lidar technology in ADAS applications, in autonomous vehicle applications,
Imricor Announces First Cases Successfully Performed at Heart Center Dresden5.2.2020 17:40:00 CET | Press release
Imricor Medical Systems, Inc. (Company or Imricor) (ASX:IMR) is pleased to announce the first procedures using the Company’s products following CE mark of the Vision-MR Ablation Catheter have been performed at the Heart Center Dresden. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200205005672/en/ Drs. Christopher Piorkowski (left) and Thomas Gaspar (right) performing iCMR ablations at the Heart Center Dresden (Photo: Heart Center Dresden). Three procedures were successfully performed over two days by Dr. Christopher Piorkowski and Dr. Thomas Gaspar (https://www.herzzentrum-dresden.com/rhythmologie/unser-team/). Additional physicians at the Heart Center Dresden will also begin performing procedures immediately. These procedures mark the first iCMR ablations anywhere in the world to ever be performed outside of a clinical trial. They were performed in an iCMR lab fitted with a Siemens 1.5T MAGNETOM Aera MR scanner. “It’s ano
VALBIOTIS Announces a Global Strategic Partnership With NESTLÉ HEALTH SCIENCE For the Development and Commercialization of TOTUM-63, a Plant-derived Active Substance With Clinically Demonstrated Metabolic Health Benefits on Prediabetics5.2.2020 17:30:00 CET | Press release
Regulatory News: VALBIOTIS (Paris:ALVAL) (FR0013254851 – ALVAL / PEA/SME eligible ), a Research & Development company committed to scientific innovation for preventing and combating metabolic diseases, announces the signature of a global long-term strategic partnership with Nestlé Health Science for the development and commercialization of TOTUM-63, an innovative and patented combination of 5 plant extracts specifically designed to reduce the risk of developing Type 2 Diabetes on prediabetics. Sébastien PELTIER, CEO of VALBIOTIS states: “Nestlé Health Science is an ideal strategic partner for VALBIOTIS. Its global reach, strategic intent to develop science-based nutritional health solutions and focus on fighting metabolic disorders like diabetes will be instrumental to TOTUM-63's worldwide commercialization success. We are excited about the opportunity that this deal brings to VALBIOTIS and to the many millions of people around the world at risk of becoming Type 2 diabetics. This trans
Skilling lanserar branschens första sömlösa integration med Spotwares cTrader, inriktad på erfarna handlare.30.1.2020 08:00:00 CET | Press release
Forex- och CFD-handelsplattformen Skilling annonserade förra veckan lanseringen av branschens första sömlösa integration med Spotwares cTrader - en unik funktion som internt kallas Ett konto. Två plattformar. Detta pressmeddelande använder multimedia. Se den fullständiga versionen här: https://www.businesswire.com/news/home/20200130005070/sv/ Skilling lanserar branschens första sömlösa integration med Spotwares cTrader. (Photo: Business Wire) Funktionen gör att Skilling kan erbjuda sina kunder sömlös handel över två olika handelsplattformar på ett enda Skilling-konto utan att behöva finansiera och hantera två olika konton separat. ”Enkelt uttryckt ville vi ge våra kunder valet vilken handelsplattform de kan använda - vår egen Skilling Trader eller en mer avancerad klientorienterad cTrader. Problemet du har med andra mäklare som erbjuder flera plattformar är behovet av att finansiera två konton separat, upprätthålla sunda marginaler på två olika konton och också ha två uppsättningar av
inRiver utser Per-Olof Schroeder till ny VD29.1.2020 16:43:00 CET | Press release
inRiver, som levererar SaaS-baserade lösningar för Product Information Management (PIM), meddelar att styrelsen idag har utsett Per-Olof Schroeder till ny VD för bolaget. Per-Olof tillträder sin tjänst med omedelbar verkan. “Styrelsen tackar Thor Johnson för hans ledning av bolaget under de två senaste åren, och är glada över att välkomna Per-Olof som vår nye VD," säger inRivers styrelseordförande, Jorgen Smidt. "Per-Olof har en påvisad erfarenhet av att mycket framgångsrikt leda utveckling av SaaS Computing Services, molnbaserade tjänster, och samtidigt leverera stark tillväxt både gällande intäkter, lönsamhet och marknadsandelar. I sin roll som bolagets VD kommer han fortsätta att utveckla inRivers mission att hjälpa våra kunder bli bäst på att marknadsföra och sälja sina produkter." Schroeder har mer än 25 års erfarenhet av ledande befattningar inom IT-branchen, inklusive 15 år på Microsoft, varav sju år som chef över Microsofts Productivity & Business Process Business Group i väste
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.Visit our pressroom